Enhanced blood–brain barrier penetration and glioma therapy mediated by a new peptide modified gene delivery system

Yao, Hui; Wang, Kaiyuan; Wang, Yi; Wang, Shanshan; Li, Jianfeng; Lou, Jinning; Ye, Liya; Yan, Xueying; Lu, Weiyue; Huang, Rongqin

doi:10.1016/j.biomaterials.2014.10.034

Cited by 97 publications

(54 citation statements)

References 40 publications

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“…Recently, Wang et al . and Yao et al . constructed a series of non‐viral gene vectors (such as cell‐penetrating peptide‐modified nanoparticles and targeting ligand‐modified nanoparticles) to package plasmid DNA encoding ING4.…”

Section: Therapeutic Genes For Cns Diseasesmentioning

confidence: 99%

“…constructed a series of non‐viral gene vectors (such as cell‐penetrating peptide‐modified nanoparticles and targeting ligand‐modified nanoparticles) to package plasmid DNA encoding ING4. In one study, they explored a novel cell‐penetrating peptide (LIM kinase 2 nucleolar translocation signal peptide, LNP)‐modified dendrigraft poly‐ l ‐lysines (DGL) dendrimer as a gene vector and plasmid DNA encoding ING4 as a therapeutic gene, aiming to achieve DGL‐PEG‐LNP/DNA nanoparticles. The DGL‐PEG‐LNP/DNA nanoparticles were shown to possess excellent BBB‐crossing efficiency and an anti‐glioma effect both in vitro and in vivo .…”

Section: Therapeutic Genes For Cns Diseasesmentioning

confidence: 99%

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Non‐viral nucleic acid delivery to the central nervous system and brain tumors

Wang

Huang

2019

The Journal of Gene Medicine

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Gene therapy is a rapidly emerging remedial route for many serious incurable diseases, such as central nervous system (CNS) diseases. Currently, nucleic acid medicines, including DNAs encoding therapeutic or destructive proteins, small interfering RNAs or microRNAs, have been successfully delivered to the CNS with gene delivery vectors using various routes of administration and have subsequently exhibited remarkable therapeutic efficiency. Among these vectors, non‐viral vectors are favorable for delivering genes into the CNS as a result of their many special characteristics, such as low toxicity and pre‐existing immunogenicity, high gene loading efficiency and easy surface modification. In this review, we highlight the main types of therapeutic genes that have been applied in the therapy of CNS diseases and then outline non‐viral gene delivery vectors.

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Section: Therapeutic Genes For Cns Diseasesmentioning

confidence: 99%

Section: Therapeutic Genes For Cns Diseasesmentioning

confidence: 99%

Non‐viral nucleic acid delivery to the central nervous system and brain tumors

Wang

Huang

2019

The Journal of Gene Medicine

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“…The RVG peptide can also modulate the accumulation of larger vehicles and has delivered macro-structures such as PAMAM dendrimers (Liu et al, 2009), liposomes (Pulford et al, 2010), chitosan nanoparticles (Gao et al, 2014), poly(mannitol- co -PEI) complexes (Park et al, 2015) and exosomes (Alvarez-Erviti et al, 2011) across the BBB and into the brain parenchyma. Other targeting agents have included: angiopep, a peptide that binds to low-density lipoprotein receptor-related protein-1 (Ke et al, 2009); lactoferrin, an iron-binding protein of the transferrin family (Huang et al, 2008, 2010); leptin, a peptide that binds to leptin receptor in different parts of the brain (Liu et al, 2010); chlorotoxin, a scorpion-derived venom that is a specific marker for gliomas (Costa et al, 2013); TGN peptide, a BBB targeting peptide isolated by phage display (Qian et al, 2013); and LIMK2 NoLs peptide, a nucleolar translocation signal sequence derived from the LIM Kinase 2 protein (Yao et al, 2015). Collectively, these targeting agents have shown to facilitate the accumulation of PEGylated PAMAM dendrimers, lysine dendrimers, liposomes, and polymeric polyplexes in the brain.…”

Section: Systemic Deliverymentioning

confidence: 99%

Non-Viral Nucleic Acid Delivery Strategies to the Central Nervous System

Tan

Sellers

Pham

et al. 2016

Front. Mol. Neurosci.

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With an increased prevalence and understanding of central nervous system (CNS) injuries and neurological disorders, nucleic acid therapies are gaining promise as a way to regenerate lost neurons or halt disease progression. While more viral vectors have been used clinically as tools for gene delivery, non-viral vectors are gaining interest due to lower safety concerns and the ability to deliver all types of nucleic acids. Nevertheless, there are still a number of barriers to nucleic acid delivery. In this focused review, we explore the in vivo challenges hindering non-viral nucleic acid delivery to the CNS and the strategies and vehicles used to overcome them. Advantages and disadvantages of different routes of administration including: systemic injection, cerebrospinal fluid injection, intraparenchymal injection and peripheral administration are discussed. Non-viral vehicles and treatment strategies that have overcome delivery barriers and demonstrated in vivo gene transfer to the CNS are presented. These approaches can be used as guidelines in developing synthetic gene delivery vectors for CNS applications and will ultimately bring non-viral vectors closer to clinical application.

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“…It has also become one of the most promising strategies to achieve non-invasive and targeted CNS gene delivery [6,7,[11][12][13]. Ultrasound-mediated MB destruction has the potential to open the BBB tight junctions and trigger therapeutic agent deposition at specific sites with non-invasive sonication [14,15].…”

Section: Introductionmentioning

confidence: 99%

“…To deliver therapeutic genes into the CNS, the first obstacle to overcome is the blood-brain barrier (BBB), which effectively blocks delivery to the brain [6,7]. The BBB is formed by the tight junctions between the endothelial cells responsibly for the barrier function, preventing uptake of most therapeutic agents into the brain.…”

Section: Introductionmentioning

confidence: 99%